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Global Haemostatic Methods Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors

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ClinicalTrials.gov Identifier: NCT02453542
Recruitment Status : Recruiting
First Posted : May 25, 2015
Last Update Posted : August 9, 2018
Sponsor:
Information provided by (Responsible Party):
Roza Chaireti, Karolinska Institutet

Brief Summary:

Background

The treatment of haemophilia A and B has been revolutionized by the use of factor concentrate, both as prophylaxis and to treat bleeding episodes (on-demand treatment). However, despite its advantages, repeated treatment with factor concentrate can lead to development of inhibitors (antibodies) towards the coagulation factor in the concentrate. Another patient group in which the bleeding symptoms are difficult to treat because of inhibitors towards coagulation factors, most commonly FVIII, is patients with acquired haemophilia. Patients with high antibody titers exhibit a deficient or no response to factor concentrates and usually need treatment with bypassing agents, namely factor eight inhibitor bypassing agent (FEIBA®, Baxter) och recombinant activated factor VII (rFVIIa, Novo-Seven®, Novo Nordisk). The effect of the treatment cannot be accurately monitored by traditional coagulation tests.

The aim of the study is to evaluate the utility of the global haemostatic methods in patients with haemophilia with inhibitors. The objective is to improve the monitoring of the treatment effect and thus increase the safety of the patient and the effectiveness of the treatment.

Patients and methods

Patients

The primary cohort will consist of fifteen patients with inherited haemophilia with inhibitors as well as five adult patients with acquired haemophilia who are followed up at the Coagulation Department of the Karolinska University Hospital, Stockholm, Sweden.

Blood samples will be collected from those patients at specific time points (see Design of the study) during the course of two years (for each patient). The treatment (type, dose, duration) will be determined by the treating physician.

Methods (selection)

  • Thrombin generation (Calibrated Automated Thrombogram, CAT® and a commercial kit from Siemens®).
  • Overall haemostatic potential (OHP)

Design of the study

Timeframe for blood sampling: i) baseline (inclusion in the study), and ii) prior and after administration of bypassing agents to either treat bleeding symptoms or before an invasive procedure or as prophylaxis.

Data analysis

The variations in coagulation markers measured as described above (Methods) will be associated to the clinical symptoms (bleeding), the level of coagulation factors (if measurable) and the titers of the inhibitors.


Condition or disease
Hemophilia

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 20 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 2 Years
Official Title: Global Haemostatic Methods to Measure the Treatment Effect Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors
Study Start Date : March 2015
Estimated Primary Completion Date : June 2019
Estimated Study Completion Date : August 2019





Primary Outcome Measures :
  1. Changes in coagulation markers, such as thrombin generation markers (Endogenous Thrombin Potential in nano molar thrombin*minute and peak thrombin in nano molar and fibrin aggregation curves, following administration of bypassing agents.) composite [ Time Frame: participants will be followed up for up to 2 years following inclusion ]

Biospecimen Retention:   Samples Without DNA
whole blood


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Ages Eligible for Study:   7 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

Fifteen patients (adults and children) with hereditary haemophilia with inhibitors.

Five patients (adults) with acquired haemophilia

Criteria

Inclusion Criteria:

  • informed consent
  • meets the study population description

Exclusion Criteria:

  • no informed consent age<7 years

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02453542


Contacts
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Contact: Roza Chaireti, MD, PhD +46 738 5170974 roza.chaireti@ki.se
Contact: Jovan Antovic, MD, Assoc Prof +46 8 517 75637 jovan.antovic@ki.se

Locations
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Sweden
Karolinska University Hospital Recruiting
Solna, Stockholm, Sweden, 17176
Contact: Roza Chaireti, MD, PhD    +46 738517974    roza.chaireti@ki.se   
Contact: Margareta Holmström, MD, Assoc Prof    +46 8 5177 0000    margareta.holmstrom@ki.se   
Sponsors and Collaborators
Karolinska Institutet
Investigators
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Principal Investigator: Roza Chaireti, MD, PhD Karolinska Institutet

Additional Information:

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Responsible Party: Roza Chaireti, MD, PhD, Karolinska Institutet
ClinicalTrials.gov Identifier: NCT02453542     History of Changes
Other Study ID Numbers: 202100-2973
First Posted: May 25, 2015    Key Record Dates
Last Update Posted: August 9, 2018
Last Verified: August 2018

Keywords provided by Roza Chaireti, Karolinska Institutet:
hemophilia
inhibitors
thrombin

Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Hemostatics
Coagulants